Guangzhou First People’s Hospital Successfully Administers Southern China’s First Gaucher Disease Gene Therapy

GUANGZHOU, China – March 3, 2025 – Guangzhou First People’s Hospital has successfully administered a gene therapy to a patient suffering from the rare genetic disorder Gaucher disease. This achievement, led by Dr. Wang Shunqing of the Hematology Department, represents a new milestone for the hospital’s Cell and Gene Therapy (CGT) sub-specialty and signals a new phase for the gene therapy field in Southern China.

The patient, who had been living with Gaucher disease for 12 years, had long relied on expensive enzyme replacement therapy (ERT) for disease management. After learning about the clinical trial for LY-M001 injection, the patient was enrolled and became the first in Southern China to receive the gene therapy. According to Dr. Yu-ping Zhang, Director of Hematology at Nansha Hospital, the patient’s condition is currently stable, and the medical team is optimistic about the long-term efficacy.

Gaucher disease (GD) is an autosomal recessive genetic disorder caused by mutations in the GBA1 gene, leading to the accumulation of metabolic substrates that cause symptoms like an enlarged liver and spleen, anemia, and bone pain. Dr. Jinqi Huang, Director of Hematology at Guangzhou First People’s Hospital, explained that while traditional treatments require lifelong medication, LY-M001 injection is designed as a “one-time, lifelong” treatment. It utilizes a recombinant adeno-associated virus (rAAV) vector to deliver a functional GBA1 gene to target organs, enabling long-term, stable expression of the necessary enzyme to degrade harmful substances.

Developed by Lingyi (Hangzhou) Biotechnology Co., Ltd., the LY-M001 injection holds independent intellectual property rights. It received dual Investigational New Drug (IND) approvals from China’s NMPA and the U.S. FDA in January 2024 and was also granted FDA Orphan Drug and Rare Pediatric Disease designations.

The successful treatment is a direct result of a collaborative innovation model involving government, industry, academia, and research institutions. The project benefited from the “pilot policies” of the Nansha Free Trade Zone and was a joint effort between Guangzhou First People’s Hospital, Lingyi Biotech, and Guangdong Medical Valley. This initiative underscores how the region is leveraging policy advantages to accelerate the translation of gene therapy technologies from the lab to the clinic.

PackGene Biotech is proud to have supported the advancement of this gene therapy in Guangzhou, providing high-quality CMC (Chemistry, Manufacturing, and Controls) services for the project. We are committed to continuing our focus on gene therapy, offering one-stop CMC solutions to support more innovative projects in the future.

About Guangzhou First People’s Hospital

Guangzhou First People’s Hospital is a large-scale Grade-A tertiary comprehensive hospital. Its hematology department is a leader in the diagnosis and treatment of rare diseases, with its CGT sub-specialty dedicated to developing and implementing innovative therapies for genetic diseases.